UCLA researchers use lipid nanoparticles to insert full CFTR gene into airway cells, restoring function and opening new ...

Researchers engineered lipid nanoparticles to deliver a full CFTR gene into human airway cells, restoring near-normal ...

Even well-studied proteins can reveal surprises. University of Iowa scientists have discovered a new enzyme activity for the cystic fibrosis transmembrane conductance regulator (CFTR). CFTR is the ...

Cystic fibrosis (CF) is the most common inherited metabolic disease in Europe. More than 8,000 people in Germany are affected ...

UCLA researchers have developed a lipid nanoparticle-based gene-editing approach capable of inserting an entire healthy gene ...

Scientists at St. Jude Children's Research Hospital and Rockefeller University have combined their expertise to gain a better understanding of the cystic fibrosis transmembrane conductance regulator ...

The cystic fibrosis transmembrane conductance regulator has been studied for years but the new efforts have yielded important insights. Scientists at St. Jude Children's Research Hospital and ...

The drug is a small molecule with a novel mechanism of action, say David Bedwell, Ph.D., and Steven Rowe, M.D., MSPH, co-senior authors. Bedwell is professor and chair of the University of Alabama at ...

Fluorescence microscope image of cells in the lung’s blood vessels. The study has shown for the first time that these cells possess a CFTR chloride channel (green), and that lung inflammation-induced ...

Protein folding diseases, from Alzheimer's to Gaucher's, may one day be treated by a unique class of protein corrector molecules that are already helping manage cystic fibrosis. Protein misfolding is ...